L-Arginine and Sickle Cell Disease

NCT01142219 | Completed Phase 3 DMC

• 1 other identifier: 04-487 cep@hcpa.ufrgs.br
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects.

Conditions

Sickle Cell Disease

Keywords

sickle cell disease; L-arginine; hemolysis; nitric oxide; pulmonary arterial hypertension

Outcome Measures

Primary Outcomes (1)

• Tricuspid regurgitant jet velocity >2.5 m/s

  Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.

  6 months

Secondary Outcomes (1)

• Lactate dehydrogenase levels

  6 months

Study Arms (1)

L-arginine

EXPERIMENTAL

0.1g/kg/day for 6 months

Drug: L-arginine; Drug: Placebo

Interventions

L-arginine DRUG

L-arginine was administered orally at a dose of 0.1g/kg/day during 6 months.

L-arginine

Placebo DRUG

Mannitol was administered orally at a dose of 01 g/kg/day for six months.

L-arginine

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis
• Genotypes SS, SC and Sβ thalassemia
• Age \> 1 year

You may not qualify if:

• Liver dysfunction (alanine aminotransferase ALT \> 3 times the normal levels
• Renal dysfunction (creatinine \> twice the normal levels)
• Increase in methemoglobin levels (\> 5 times the normal levels)
• History of recent stroke (\< 1 month) and priapism
• Pregnancy
• Allergy to L-arginine
• Use of sildenafil, calcium channel blockers, nitroglycerin or other nitrates

Sponsors & Collaborators

• Hospital de Clinicas de Porto Alegre: lead
• Fundo de Incentivo à Pesquisa do Hospital de Clinicas de Porto Alegre (FIPE): collaborator
• Associação de Amigos da Hematologia (HEMOAMIGOS): collaborator
• Ajinomoto Interamericana Indústria e Comércio Ltda. (donation of L-arginine): collaborator

Study Sites (1)

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-001, Brazil

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemolysis; Pulmonary Arterial Hypertension

Interventions

Arginine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Amino Acids, Basic; Amino Acids; Amino Acids, Peptides, and Proteins; Amino Acids, Diamino; Amino Acids, Essential

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 10, 2010
• First Posted: June 11, 2010
• Study Start: September 1, 2006
• Primary Completion: January 1, 2008
• Study Completion: October 1, 2009
• Last Updated: June 11, 2010

Record last verified: 2010-06

Locations

BR (1)